This is a prospective, single-arm study to investigate the efficacy and safety of the combination of fixed low-dose eltrombopag plus recombinant human thrombopoietin (rhTPO) as treatment for corticosteroid-resistant or relapsed immune thrombocytopenia (ITP) patients during the COVID-19 pandemic.
Eltrombopag, a small molecule agonist of thrombopoietin receptor (TPO-RA), was recommended as the subsequent treatment for ITP patients, which also already showed robust efficacy.Recombinant human thrombopoietin (rhTPO) is a full-length glycosylated-TPO produced by Chinese hamster ovary cells, which showed its effectiveness in ITP in a variety of studies. Both eltrombopag and rhTPO demonstrated good safety in ITP patients. Because of their non-immunosuppressive nature, both of them serve as a reasonable choice during the global COVID-19 pandemic. Since they increase the number of platelets through different mechanisms, and previous studies demonstrated that they might exert synergic effect. The investigators hypothesized that the combination of these two agents could be a promising option for treatment of corticosteroid-resistant or relapsed ITP patients.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Fixed dose of eltrombopag oral 25mg daily
Rh-TPO 300U/kg subcutaneous injection once daily for 7 consecutive days, followed by a tapering dose in maintenance therapy.
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, China
RECRUITINGComplete response
A complete response (CR) was defined as a sustained (≥ 3 months) platelet count ≥ 100×10\^9/L.
Time frame: 6 weeks
Response
A response (R) was defined as a sustained (≥ 3 months) platelet count ≥ 30×10\^9/L without recurrence of thrombocytopenia.
Time frame: 6 weeks
No response
No response (NR) was defined as platelet count \< 30 × 10\^9/L or a less than two fold increase in platelet count from baseline or the presence of bleeding. Platelet count must be measured on two occasions more than a day apart.
Time frame: 6 weeks
Relapses
A relapses was defined as platelet count falls below 30×10\^9/L or bleeding accrues after achieving R or CR.
Time frame: 6 weeks
Early response
Early response was defined as the attainment of a platelet count ≥ 30 × 10⁹ and at least a doubling of baseline platelet count at 1 week.
Time frame: 7 days
Initial response
Initial treatment was defined as the attainment of a platelet count ≥ 30 × 10⁹ and at least a doubling of baseline platelet count at 1 month.
Time frame: 1 month
TOR (time to response)
The time to achieve platelet count ≥ 30×10\^9/L and at least 2-fold increase of the baseline count and absence of bleeding since start of treatment.
Time frame: 6 weeks
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DOR (duration of response)
The duration of achieve platelet count ≥ 30×10\^9/L and at least 2-fold increase of the baseline count and absence of bleeding since start of treatment.
Time frame: 6 weeks
Treatments associated adverse events
All patients were assessed for safety every week during the first 8 weeks of treatment, and at 2-week intervals thereafter. Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time frame: 6 weeks
Reduction in bleeding symptoms
Changes of bleeding after treatment. Bleeding was defined in accordance with the WHO bleeding scale (0, no bleeding; 1, petechiae; 2, mild blood loss; 3, gross blood loss; and 4, debilitating blood loss).
Time frame: 6 weeks